Use of Real-World Data Sources for Canadian Drug Pricing and Reimbursement Decisions: Stakeholder Views and Lessons for Other Countries.

BACKGROUND: Canada has a long history of the use of clinical evidence to support healthcare decision making. Given improvements in data holdings and analytic capacity in Canada and stakeholder interest, the purpose of this study is to reflect on perceptions of the value of real-world evidence in pricing and reimbursement decisions, barriers to its optimal use in pricing and reimbursement, current initiatives that may lead to its increased use, and what role the pharmaceutical industry may play in this.Methods/ResultsTo capture stakeholder perceptions, ninety-one participants identified as key stakeholders were identified according to background roles and geography and invited to participate in four round table discussions conducted under Chatham House rule. Important themes emerging from these discussions included: (i) the need to understand what "real world" evidence means; (ii) barriers to using real world evidence from differences in access, governance, inter-operability, system structures, expertise, and quality across Canadian health systems; (iii) differing views on industry's role. CONCLUSIONS: The use of real-world data in Canada to inform pricing and reimbursement decisions is far from routine but nascent and slowly increasing. Barriers, including interoperability concerns, may also apply to other federated health systems that need to focus on the networking of healthcare administrative data across provincial jurisdictional boundaries. There also appears to be a desire to see better use of pragmatic trials linked to these administrative data sets. Emerging initiatives are under way to use real world evidence more broadly, and include identification of common data elements and approaches to networking data.

The Structural Genomics Consortium: A Knowledge Platform for Drug Discovery: A Summary.

The Structural Genomics Consortium (SGC) supports drug discovery efforts through a unique, open access model of public-private collaboration. This study presents the results of an independent evaluation of the Structural Genomics Consortium, conducted by RAND Europe with the Institute on Governance. The evaluation aimed to establish the role of the SGC within the wider drug discovery and PPP landscape, assessing the merits of the SGC open access model relative to alternative models of funding R&D in this space, as well as the key trends and opportunities in the external environment that may impact on the future of the SGC. It also established the incentives and disincentives for investment, strengths and weaknesses of the SGC's model, and the opportunities and threats the SGC will face in the future. This enabled us to assess the most convincing arguments for funding the SGC at present; important trade-offs or limitations that should be addressed in moving towards the next funding phase; and whether funders are anticipating changes either to the SGC or the wider PPP landscape. Finally, we undertook a quantitative analysis to ascertain what judgements can be made about the SGC's past and current performance track record, before unpacking the role of the external environment and particular actors within the SGC in developing scenarios for the future.

Bibliometrics as a Tool for Supporting Prospective R&D Decision-Making in the Health Sciences: Strengths, Weaknesses and Options for Future Development.

Bibliometric analysis is an increasingly important part of a broader "toolbox" of evaluation methods available to research and development (R&D) policymakers to support decision-making. In the US, UK and Australia, for example, there is evidence of gradual convergence over the past ten years towards a model of university research assessment and ranking incorporating the use of bibliometric measures. In Britain, the Department of Health (England) has shown growing interest in using bibliometric analysis to support prospective R&D decision-making, and has engaged RAND Europe's expertise in this area through a number of exercises since 2005. These range from the macro-level selection of potentially high impact institutions, to micro-level selection of high impact individuals for the National Institute for Health Research's faculty of researchers. The aim of this study is to create an accessible, "beginner's guide" to bibliometric theory and application in the area of health R&D decision-making. The study also aims to identify future directions and possible next steps in this area, based on RAND Europe's work with the Department of Health to date. It is targeted at a range of audiences, and will be of interest to health and biomedical researchers, as well as R&D decision-makers in the UK and elsewhere. The study was completed with funding support from RAND Europe's Health R&D Policy Research Unit with the Department of Health.